Deflazacort (Emflaza) is a corticosteroid and has anti-inflammatory and immune-modulatory effects. It is used to treat patients with Duchenne muscular dystrophy who are at least 5 years of age or older.

Deflazacort Uses:

• Duchenne muscular dystrophy:

  • Treatment of Duchenne muscular dystrophy (DMD) in patients 5 years and older

Deflazacort (Emflaza) Dose in Adults

Deflazacort (Emflaza) Dose in the treatment of Duchenne muscular dystrophy:

• Oral: Usual dose:
  • 0.9 mg/kg one time a day.
• Note: Round up to nearest possible dose when using tablets; round up to the nearest tenth of an mL when using suspension.

• Concomitant moderate or strong CYP3A4 Inhibitors (eg, clarithromycin, fluconazole, diltiazem, verapamil):

  • Reduce deflazacort dose to 1/3 rd of the usual dose.

Deflazacort (Emflaza) Dose in Childrens

Deflazacort (Emflaza) Dose in the treatment of Duchenne muscular dystrophy:

• Male children ≥5 years and Adolescents:

  • Oral: About 0.9 mg/kg/dose one time a day;
  • The doses should be rounded based on product formulation:
    • Oral suspension: Round dose up to nearest 0.1 mL (a tenth of an mL);
    • Tablets: Round up to the nearest possible dose based on tablet strengths, any combination of available tablets strengths may be used

• Dosing adjustment for concomitant therapy with moderate or strong CYP3A4 inhibitors (eg, clarithromycin, fluconazole, diltiazem, verapamil):

  • Male children ≥5 years and Adolescents:
    • Reduce deflazacort dose to 1/3 rd of the usual dose

Pregnancy Risk Category: N

• Deflazacort enters into the placenta.
• Maternal use has been linked to oral clefts, intrauterine restriction and reduced birth weight.
• After maternal use of corticosteroids during the pregnancy, hypoadrenalism can occur in newborns.

Use during breastfeeding:

• Breast milk contains corticosteroids.
• We have not found any data related to deflazacort.
• According to the manufacturer, it is important to consider whether to discontinue or continue breastfeeding during treatment.
• This decision will be based on the risks of infant exposure, the benefits to the infant and the benefits to the mother.

Dose in Kidney Disease:

No dosage modification required; use with caution.

Dose in Liver disease:

• Mild to moderate impairment:

  • No dosage modification required.

• Severe impairment:

  • There is no dosage modification given in the manufacturer’s labeling (has not been studied).

Common Side Effects of Deflazacort (Emflaza):

• Dermatologic:

  • Erythema

• Endocrine & metabolic:

  • Cushingoid appearance
  • Hirsutism
  • Weight gain
  • Obesity

• Gastrointestinal:

  • Abdominal pain
  • Increased appetite

• Genitourinary:

  • Pollakiuria

• Respiratory:

  • Cough
  • Upper respiratory tract infection

Less Common Side Effects of Deflazacort (Emflaza):

• Cardiovascular:

  • Cardiac Arrhythmia

• Central Nervous System:

  • Irritability
  • Abnormal Behavior
  • Psychomotor Agitation
  • Aggressive Behavior
  • Depression
  • Dizziness
  • Emotional Disturbance
  • Emotional Lability
  • Heat Exhaustion
  • Hypertonia
  • Insomnia
  • Mood Changes
  • Sleep Disorder

• Dermatologic:

  • Skin Rash
  • Atrophic Striae
  • Acneiform Eruption
  • Acne Vulgaris
  • Alopecia
  • Impetigo

• Endocrine & Metabolic:

  • Glycosuria
  • Hot Flash
  • Increased Thirst

• Gastrointestinal:

  • Constipation
  • Abdominal Distress
  • Nausea
  • Dyspepsia
  • Gastrointestinal Disease

• Genitourinary:

  • Dysuria
  • Testicular Pain
  • Urinary Tract Infection
  • Urine Discoloration

• Hematologic & Oncologic:

  • Bruise

• Infection:

  • Influenza
  • Tooth Abscess
  • Viral Infection

• Neuromuscular & Skeletal:

  • Back Pain
  • Back Injury
  • Limb Pain
  • Muscle Spasm
  • Myalgia
  • Neck Pain

• Ophthalmic:

  • Hordeolum
  • Increased Lacrimation

• Otic:

  • Otitis Externa

• Respiratory:

  • Nasopharyngitis
  • Rhinorrhea
  • Epistaxis
  • Hypoventilation
  • Pharyngitis

• Miscellaneous:

  • Fever
  • Accidental Injury
  • Mass

Frequency of side effects not defined.

• Central nervous system:

  • Myasthenia (associated with long-term use)

• Neuromuscular & skeletal:

  • Bone fracture (long bones including the fibula as well as greenstick fractures)
  • Decreased bone mineral density
  • Osteopenia (associated with long term use)
  • Tendon disease (associated with long-term use)

Contraindications to Deflazacort (Emflaza):

Sensitivity towards deflazacort and any component of the formulation

Warnings and precautions

• Suppression of the adrenals:

  • May cause hypercortisolism or suppression of the hypothalamic-pituitary-adrenal (HPA) axis. Suppression of the HPA axis may result in adrenal crisis.
  • It is important to withdraw slowly and carefully from corticosteroid use.

• Anaphylaxis

  • Rare cases of anaphylaxis have been reported in patients who received corticosteroids.

• Immunosuppression:

  • Corticosteroids can increase the risk of secondary infections, activate suppressed infections, cloak serious infection (including fungal infections), prolong and worsen existing infections, and limit or inhibit response to inactivated or killed vaccines.
  • Avoid exposure to measles or chickenpox.
  • Patients with underlying tuberculosis or TB reactivity need to be closely monitored. Limit active TB use (only when combined with antituberculosis therapy).
  • Patients with active ocular herpes easyx should not be given this medication. Patients who are hepatitis B-infected can experience a recurrence of hepatitis B.
  • Before starting corticosteroids, it is important to rule out amebiasis in patients who have recently traveled to tropical climates or unexplained diarrhea.
  • Patients with Strongyloides infections should be vigilant; cases of hyperinfection, spread, and even death have been reported.

• Kaposi Sarcoma:

  • Kaposi Sarcoma has been linked to long-term corticosteroids therapy. If this is the case, it should be terminated.

• Myopathy

  • High dose corticosteroids have been associated with severe myopathy, most commonly in patients with neuromuscular transmit disorders. This may include ocular or respiratory muscles. Monitor creatinine kinase for signs of recovery.

• Ocular effects

  • Extended use can lead to posterior subcapsular cataracts, increased intraocular pressure and nerve damage.
  • Routine eye exams are a good idea for chronic users.

• Psychiatric disorders:

  • Corticosteroid abuse can cause psychotic disturbances such as depression, euphoria and insomnia.
  • Corticosteroid treatment can worsen pre-existing mental conditions.

• Reactions to skin:

  • Within 8 weeks of starting therapy, toxic epidermal necrolysis was reported. Stop using the medication immediately if you notice a rash.

• Events that are thromboembolic:

  • Higher cumulative corticosteroids doses are associated with thromboembolism risk
  • Patients with thromboembolic disorders or a history of them should be cautious.

• Cardiovascular disease

  • Patients with heart disease and/or hypertension should be cautious; fluid retention, electrolyte disturbances and hypertension have been reported.
  • Follow myocardial injury with care Corticosteroids have been shown to cause myocardial tears/ rupture.

• Diabetes:

  • Patients with diabetes mellitus should be careful when using this product. It may alter glucose production/regulation leading to hyperglycemia.

• Gastrointestinal Disease:

  • Patients with GI disorders (diverticulitis fresh intestinal anastomoses ulcerative colitis active or suppressed peptic ulcer, abscess or other pyogenic infection) should be cautious.
  • If there is a possibility of perforation, abscess or any other pyogenic infection, avoid use.

• Hepatic impairment

  • Patients with severe hepatic injury should be cautious.

• Myasthenia gravis:

  • Patients with myasthenia Gravis should be careful. Especially early treatment with corticosteroids can lead to aggravation of symptoms.

• Osteoporosis

  • Patients with osteoporosis or at high risk of developing it should be treated carefully.
  • Corticosteroids are associated with bone loss, osteoporotic fractures and avascular necrosis when taken in high doses or for long periods of time.

• Pheochromocytoma:

  • Patients with pheochromocytoma should be cautious. Corticosteroids have been used to treat pheochromocytoma crises, which can prove fatal.

• Renal impairment

  • Avoid kidney damage. Fluid retention may occur.

• Seizure disorders:

  • Patients with seizure disorders should be cautious.

• Thyroid disease:

  • Modifications to dosage may be required for thyroid changes.
  • Hyperthyroid patients have a higher metabolic clearance of corticosteroids than those with hypothyroidism.

Deflazacort: Drug Interaction

Note: Drug Interaction Categories:

• Risk Factor C: Monitor When Using Combination
• Risk Factor D: Consider Treatment Modification
• Risk Factor X: Avoid Concomitant Use

Monitoring parameters:

Blood pressure, blood glucose, and electrolytes.

After extended use:

• Bone mass density, assess HPA axis suppression (eg, ACTH stimulation test, morning plasma cortisol test, and urinary free cortisol test), growth in children, signs, and symptoms of infection, cataract formation, and intraocular pressure.

How to administer Deflazacort (Emflaza)?

Oral: Administer with or without food.

Tablets:

• Administer whole or may crush and mix with applesauce (administer applesauce mixture immediately).

Suspension:

• Shake well; measure prescribed dose with provided dispenser, mix thoroughly with 3 to 4 ounces of juice or milk, and administer immediately. Do not mix or administer with grapefruit juice.

Mechanism of action of Deflazacort (Emflaza):

• Deflazacort, a corticosteroid drug prodrug, is available. Its active metabolite 21-desDFZ acts on the glucocorticoid receptor to produce anti-inflammatory, immunosuppressive and other effects.
• It is not known how deflazacort works in Duchenne muscular dystrophy patients.

Protein binding

• About 40% of the population is protein-bound.

Metabolism:

• It is quickly converted by esterases into 21-desDFZ (active metabolite); 21–desDFZ is metabolized by CYP3A4 into several metabolites (inactive).

Time to peak, serum:

• 1 hour (range: 0.25 to 2 hours);
• The time to peak is delayed by one hour with a high-fat meal

Excretion:

• Urine (~68%; 18% as 21-desDFZ)

International Brand Names of Deflazacort:

• Emflaza
• Aflazacort
• Alcoza
• Alfazcort
• Alnacort
• Arocort
• Arrumal
• Asfor
• Asodef
• Avantium
• Azacortid
• Calcorrt
• Calcort
• Carzoflep
• Clobax
• Cortiflo
• Cortimax
• Dazenar
• Defal
• Defas
• Defax
• Defcort
• Defla
• Deflaimmun
• Deflan
• Deflanil
• Deflazym
• Defzort
• Dezacor
• Dezartal
• DFZ
• Dispercort
• Flacort
• Flamirex
• Flantadin
• Flazacor
• Flazacort
• Flazal
• Landacort
• Lantadin
• Prandin
• Prism
• Refla
• Rosilan
• Servicor
• Setatrep
• Telacort
• Xalcort
• Zamen
• Zamene

Deflazacort Brand Names in Pakistan:

No Brands Available in Pakistan.